Compositions and methods for the treatment of wounds, disorders, and diseases of the skin
a technology for disorders, wounds, and skin, applied in the direction of drug compositions, enzymology, viruses, etc., can solve the problems of difficult to chew and swallow food, chronic malnutrition, and excessive scarring, and achieve the effects of increasing, increasing, and/or supplementing the formation of hydroxylysine residues, enhancing, increasing, and/or supplementing the formation of anchoring fibrils of subjects
Active Publication Date: 2017-10-12
KRYSTAL BIOTECH INC
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AI Technical Summary
Benefits of technology
The present patent relates to a method of using a pharmaceutical composition containing a recombinant herpes simplex virus genome to provide prophylactic, palliative, or therapeutic relief of wounds, disorders, or diseases of the skin in a subject. The method involves administering the pharmaceutical composition to the subject and enhancing, increasing, or supplementing the levels of certain proteins in the subject's cells. The pharmaceutical composition can be administered through various routes such as topical or transdermal application. The recombinant herpes simplex virus genome can also contain other genes or transgenes to further enhance the therapeutic effect. The use of the pharmaceutical composition can lead to the formation of anchoring fibriles and enhance the stability and strength of the skin.
Problems solved by technology
Blisters are routinely present over the whole body, including on mucous membranes (such as the lining of the mouth and digestive tract), and healing of these blisters results in extensive scarring.
Damage to the mouth and esophagus can make it difficult to chew and swallow food, leading to chronic malnutrition and slow growth.
Complications from extensive scarring can include fusion of the fingers and toes, joint deformities, and eye inflammation leading to vision loss.
Additionally, patients suffering from RDEB have a high risk of developing squamous cell carcinoma, which can be unusually aggressive in this patient population, often becoming life-threatening.
Mutations in the Col7a1 gene, and diminished levels of PLODS expression, impair the ability of Collagen alpha-1 (VII) chain protein to properly connect the epidermis to the dermis in dystrophic epidermolysis bullosa patients, leading to fragile skin.
Treatment options for epidermolysis bullosa patients are limited, and current care focuses on managing the symptoms of the disease, including providing medication to control pain and itching, administering oral antibiotics to stave off infections resulting from open wounds on the skin and mucosa, and surgical strategies to address scarring and deformities.
Because many DEB patients have multiple wounds spanning large areas of trauma-prone sites (such as the sacrum, hips, feet, lower back, and hands), any treatment involving intradermal injection would be extremely invasive, as these large wound areas would all need to be injected, likely repeatedly, although injection time intervals are unclear.
Method used
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embodiment 1
[0158]A pharmaceutical composition comprising:
[0159]a) a virus comprising a vector, wherein the vector comprises one or more transgenes encoding a polypeptide selected from the group consisting of a Collagen alpha-1 (VII) chain polypeptide, a Lysyl hydroxylase 3 polypeptide, and a chimeric polypeptide thereof; and
[0160]b) a pharmaceutically acceptable carrier.
embodiment 2
[0161]The pharmaceutical composition of embodiment 1, wherein the virus is an adenovirus, adeno-associated virus, retrovirus, lentivirus, sendai virus, herpes simplex virus, vaccinia virus, or any hybrid virus thereof.
embodiment 3
[0162]The pharmaceutical composition of embodiment 1, wherein the virus is a herpes simplex virus (HSV).
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Abstract
The present disclosure relates, in part, to pharmaceutical compositions comprising one or more polynucleotides suitable for enhancing, increasing, augmenting, and / or supplementing the levels of Collagen alpha-1 (VII) chain polypeptide and / or Lysyl hydroxylase 3 polypeptide and / or Keratin type I cytoskeletal 17 polypeptide in a subject. The present disclosure also relates, in part, to pharmaceutical compositions and methods of use for providing prophylactic, palliative, or therapeutic relief of a wound, disorder, or disease of the skin in a subject, including a subject having, or at risk of developing, one or more symptoms of epidermolysis bullosa.
Description
CROSS REFERENCE TO RELATED APPLICATIONS[0001]This application claims the priority benefit of U.S. Provisional Application Ser. No. 62 / 320,316, filed Apr. 8, 2016, which is incorporated herein by reference in its entirety.SUBMISSION OF SEQUENCE LISTING ON ASCII TEXT FILE[0002]The content of the following submission on ASCII text file is incorporated herein by reference in its entirety: a computer readable form (CRF) of the Sequence Listing (file name: 761342000100SEQLIST.txt, date recorded: Dec. 28, 2016, size: 394 KB).FIELD OF THE INVENTION[0003]The present disclosure relates, in part, to pharmaceutical compositions and methods of use for providing prophylactic, palliative, or therapeutic relief of a wound, disorder, or disease of the skin in a subject, including a subject having, or at risk of developing, one or more symptoms of epidermolysis bullosa.BACKGROUND[0004]A number of serious disease-related skin conditions are associated with one or more genetic disorders in patients suf...
Claims
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IPC IPC(8): A61K35/763A61K9/00
CPCA61K9/0014A61K35/763A61K38/1748A61K38/39A61K9/06A61K47/38A61K48/005C07K14/78C12N9/0071C12Y114/11004C12N2710/16643A61P17/00A61P17/02A61P17/04A61P17/16A61P35/00A61K38/443A61K39/12A61K2039/53C12N15/86C12N7/00C12N2710/16034
Inventor KRISHNAN, SUMAAGARWAL, POOJA
Owner KRYSTAL BIOTECH INC




